When Leola Conleys daughter was born, everybody said she looked like she was already three months old.

The girl was 8 pounds, 13 ounces, cute, with a full head of hair, said Conley, a counselor in Lafayette.

About a week after her birth, a letter arrived from the state Department of Health. A standard pinprick on the babys heel at birth showed a mutation in a gene that makes red blood cells. Usually, red blood cells are round, like a doughnut. This babys cells were shaped like a crescent moon. She had sickle cell disease, a genetic disorder that affects about one in every 365 Black babies. Those with severe cases have a life expectancy of about 45 years.

It put me in a depression like nobody's business, said Conley. It broke me, because she looked so healthy.

Now 23, Conleys daughter, Adria, guesses shes been hospitalized about 30 times. She graduated with her MBA in December but is limiting her job search to positions with good insurance close to her doctors in south Louisiana. She doesnt look sick, but unpredictable pain episodes can throw her life off track, requiring weeks of recovery.

But new gene therapies two of which will submit for FDA approval by the end of March offer what could be a life-changing alternative for sickle cell patients in Louisiana,which has the second-highest prevalence of sickle cell disease among Medicaid patients. The problem? At a predicted $1 million to $2 million for the therapy, its unclear how states will pay for it.

If just 7% of the eligible sickle cell population of Louisiana wanted the treatment, it would cost the state a predicted $31.6 million in the first year, according to an analysis published in JAMA Pediatrics by Dr. Patrick DeMartino, a physician specializing in blood diseases among children. Thats for just 17 patients.

Nobody really knows whether it's going to be three people getting the gene therapy in the first year or 15, said DeMartino. And that upper end of 15 to 20 people a year would almost certainly be very consequential for the Medicaid budget.

No one tracks how many Louisiana residents have sickle cell disease, though efforts to create a registry are underway. There are at least 3,000 people on Medicaid with sickle cell disease, according to the state. The 1,400 who were hospitalized in Louisiana in 2020 spent an average of 30 days in the hospital. About one in 10 children with sickle cell anemia will have a symptomatic stroke by age 20, and more have strokes that go undetected.

These people do not live normal lives at all, said DeMartino. The existing therapies are supportive. They don't change the underlying disease, and people still have massive amounts of disability.

Instead of easily transporting oxygen through their bodies, the warped cells become wedged in blood vessels, causing blockages and injuring the vessels, resulting in immense pain.

Have you ever felt your heart beating in your ear? It feels like that but all over your body, said Whitney Carter, a 37-year-old who works from home for an insurance company in Baton Rouge.

The sickled cells getting jammed in her blood vessels feels "like a walnut trying to fit through a straw.

Without a potential pain episode looming over Rhonda Chubes 9-year-old son, he would be able to keep up with his older brothers, play football, swim and collect rocks to his hearts content. With treatment, hed be a regular kid, said Chube, a child care assistant at a Baton Rouge hospital.

His little spirit, he wants to do more, keep going, said Chube. But his little body won't let him.

Prior to gene therapy, a bone-marrow transplant was the only thing close to a cure, but required a close genetic match, usually a sibling, resulting in a match for about 25% of patients. There is also risk that the body will reject the transplant, causing serious complications or death.

Researchers shy away from using the word cure to describe gene therapy, because its not yet clear how long its effects last since the first was given in 2017. But it has been shown to deliver a pain-free existence for trial participants for the first time in their lives.

There are two different gene therapy drugs to treat sickle cell expected to be approved soon one from Bluebird Bio and the other from Vertex and CRISPR Therapeutics.

Its highly transformative, said Dr. Julie Kanter, a hematologist at the University of Alabama at Birmingham who oversees a Bluebird trial that includes patients from Louisiana.

Whats most notable when I look at my patients is how many are now working that couldnt work before, said Kanter. I have one in nursing school, one in a full-time construction job They dont have to worry about being too far from a hospital or too far from pain medicines.

But how to deliver the medications, which will require an expensive and lengthy hospital stay not included in the drugs price tag, is something the world is trying to figure out right now, said Kanter, a New Orleans native who was previously head of the Sickle Cell Center of Southern Louisiana.

Figuring out a way to fund sickle cell treatment is a priority of the Biden administration. And Louisiana has gotten creative with paying for expensive therapies before, Dr. Joe Kanter, state health officer, pointed out. The state got unlimited access to pricey hepatitis C medication for five years for a lump sum instead of paying per dose.

I think the hepatitis C work laid a good groundwork for the feds and other states to follow if they choose to, said Kanter, who is not related to Julie Kanter.

The U.S. Department of Health and Human Services released a plan in mid-February that aims to address the sky-high cost of gene therapy. A pilot program would allow the federal government to coordinate a way to pay on behalf of states. For example, Medicaid might negotiate to pay for treatment in installments dependent on the therapy continuing to prevent pain, which has not yet been proven.

The plan will likely take years to implement.

Still, not everyone will want the therapy, which requires a long hospital stay and chemotherapy to make room for new stem cells.

For many patients, even words like transformative and life-changing can seem empty after years of mistreatment.

Every time I go to the hospital, I feel like I have to put my shield on, because Im going to battle, said Leola Conley. Who will I fight today?

Conley and her daughter have endured 11-hour waits in emergency rooms to get admitted and health care workers who dont believe her pain.

A lack of investment in treatment for sickle cell patients means that the only option for some is often opioids in an emergency room, said Dr. Jennifer Avegno, an emergency medicine physician and director of the New Orleans Health Department.

"Being on chronic opiates is really a terrible way to live," said Avegno. "We've really taken a terrible disease and made it worse in America."

In comparison to sickle cell, genetic diseases that mainly affect White people, such as cystic fibrosis, have much more investment, Avegno pointed out. A federal study found funding for cystic fibrosis was 11 times that of per-person funding for sickle cell disease, even though it impacts about one-third of the number of people.

In a world without sickle cell,Adria Conleyimagines shed move to the Netherlands, where shes read people are happy.Maybe one day, with gene therapy, that will be a reality. For now, sickle cell is calling the shots.

If gene therapy was offered as a safe and affordable option, we would run to it, said Leola Conley. Her fear is that one day, her daughter will be in pain and she wont be around to make sure she gets the right treatment.

Before I leave this earth, I hope to heck my child can be cured, said Conley. She has so much to offer the world.

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New gene therapies are a near-cure for Louisiana's sickle cell patients. But at $1-2M, who will pay for it? - NOLA.com